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Virus Therapy

SAR444836 for Phenylketonuria

Phase 1 & 2

Recruiting

Research Sponsored by Sanofi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Two plasma Phe values ≥ 600 μmol/L drawn at least 72 hours apart during the screening period while on Phe restricted diet therapy in the absence of an acute illness

Two historical plasma Phe values ≥ 600 μmol/L in the preceding 12 months while on Phe restricted diet therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to week 96

Awards & highlights

Study Summary

"This trial is testing a new gene therapy called SAR444836 for adults with phenylketonuria (PKU). The goal is to see if SAR444836 can reduce phenylalanine levels

Who is the study for?

Adults aged 18-65 with Phenylketonuria (PKU) due to PAH deficiency, who have been on a stable diet restricting phenylalanine. Participants must have had high plasma Phe levels in the past while on this diet and be willing to maintain their current diet for the trial's duration. They should not be able to bear children, have a BMI ≤ 35 kg/m2, and agree to use contraception.Check my eligibility

What is being tested?

The study tests SAR444836, a gene therapy aiming to treat PKU by introducing a functional version of the PAH enzyme via an adeno-associated virus vector. It involves one IV administration followed by monitoring over approximately two years with up to 41 visits which may include remote assessments.See study design

What are the potential side effects?

While specific side effects are not listed here, participants will be closely monitored for any adverse events following the intravenous administration of SAR444836. This includes tracking organ function and overall health throughout the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My blood tests show high phenylalanine levels twice, despite my diet.

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My blood Phe levels were high twice in the last year despite my special diet.

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I am between 18 and 65 years old and either a man or a woman who cannot become pregnant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to week 96

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to week 96

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to week 96 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of treatment-emergent adverse events (TEAEs)

Secondary outcome measures

Assessment of the duration of viral vector shedding of SAR444836 in sampling of urine, saliva, and semen at 4-week intervals following SAR444836 administration

Change from baseline in dietary protein intake at Week 24 and Week 96 or End of Study following SAR444836 administration

Change from baseline in plasma Phe: Tyr ratio at Week 24 and Week 96 or End of Study following SAR444836 administration

+5 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: SAR444836Experimental Treatment1 Intervention

Participants will receive a single dose of SAR444836 on Day 1

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor

2,164 Previous Clinical Trials

3,514,948 Total Patients Enrolled

Clinical Sciences & OperationsStudy DirectorSanofi

8 Previous Clinical Trials

4,898 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the age criterion for participating in this medical study limited to individuals below 70 years old?

"As per the trial's eligibility criteria, individuals aged 18 to 65 are eligible for enrollment."

Answered by AI

How many participants are currently undergoing treatment within this clinical trial?

"Indeed, the details on clinicaltrials.gov show that this investigation is actively seeking volunteers. Initially shared on August 7th, 2023, and last modified on February 8th, 2024, the trial aims to enroll a total of 32 participants from four distinct locations."

Answered by AI

Is the enrollment for this study currently available to prospective participants?

"Affirmative, data on clinicaltrials.gov confirms that this investigation is actively enrolling subjects. The study was initially posted on August 7th, 2023 and last revised on February 8th, 2024. Recruitment aims to enlist a total of 32 participants from four distinct sites."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

2023. "A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05972629.

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